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Nov 11, 2025 12:00 PM

CRISPR Therapeutics' Investigational Gene Therapy Shows Promising Lipid-Lowering Effects In Early Trial

On Saturday, CRISPR Therapeutics AG (NASDAQ:CRSP) shared Phase 1 data from its ongoing clinical trial evaluating CTX310, an investigational, in vivo CRISPR/Cas9 gene-editing therapy targeting ANGPTL3.

ANGPTL3, or Angiopoietin-Like 3, is a protein primarily produced in the liver that plays a major role in regulating blood lipid (fat) levels, particularly triglycerides and cholesterol.

A single-course treatment with CTX310 produced dose-dependent, durable reductions in circulating ANGPTL3 with a mean decrease from baseline of -73% (maximum -89%), a mean reduction in triglycerides (TG) of -55% (maximum -84%) and a mean reduction of low-density lipoprotein (LDL) of -49% (maximum -87%) at the highest dose.

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